Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A.

What was studied

This phase 3 study tested rurioctocog alfa pegol in severe hemophilia A. People had not been treated before. It was an open-label study with no control group, at many centres. People got the drug into a vein, to prevent or treat bleeds. The main goal was how often FVIII inhibitors developed. FVIII is the clotting factor replaced in this disease. Inhibitors can be serious. These are the final data.

What they found

146 patients enrolled and 120 got at least 1 dose. In the main group of 100, 11 developed inhibitors. 89 had 100 or more exposure days without inhibitors. The incidence was 0.110, with a range of 0.056-0.188. Bleeding rates per year were 1.7 with once-weekly prevention. They were 1.0 with more frequent prevention and 2.3 with on-demand care. 109 of 120 patients had adverse events. Serious events occurred in 47 patients.

What it means for patients

The authors say inhibitors of FVIII were uncommon in people not treated before. They say efficacy and safety results were similar to earlier reports in previously treated patients.

Why it is not certain yet

This was an uncontrolled trial. There was no comparison group. The inhibitor analysis used 100 of the 120 patients. The abstract also gives post hoc analyses. The abstract does not say how the drug compares with other clotting drugs.

Source

Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A.

We restate facts from the source record in our own words. The original wording belongs to its publisher.